Neuro-Oncology Basic Research
Nancy Ratner, PhD, is the Director of Neuro-Oncology Research in the Cancer Program at Cincinnati Children's Hospital Medical Center. Faculty members at the medical center are internationally recognized for leading basic and translational research in pediatric glial tumors. Active areas of research include:
- Molecular and cellular basis of patterning and cell-type specification
- Stem cell biology
- Signal transduction
- Transcriptional regulation
- Tumorigenesis
- Animal behavioral studies
For research in the biology and therapy of gliomas and also for tumors in patients with neurofibromatosis type 1 (NF 1), the neural tumor basic research program led by Dr. Nancy Ratner is one of the nations leading centers and federally funded (NIH and Department of Defense) research and tumor genesis through the NF gene pathway.
Dr. Ratner's work has identified novel molecular targets for therapy in these tumors and has led to the development of phase 1 clinical trials in this area. In parallel, integrated efforts by Dr. George Thomas have identified critical and key areas for cancer cell signaling through mTOR/S6K1 signaling axis in neural and other tumors.
The formation of integrated laboratory and translational programs constituting the Cincinnati Center for Neurofibromatosis Research beginning in 2005 has catalyzed the development of exciting collaborations for translational drug development. These projects are also conducted in collaboration with the University of Cincinnati Genome Research Institute's Drug Discovery Center and has used their large, chemically-diverse compound repository and new anticancer drug high throughput screening (HTS) facility.
Masato Nakafuku MD, PhD, is an Ohio Eminent Scholar, and leads the research Program in Developmental Neurobiology at Cincinnati Children's. Research in the Nakafuku laboratory centers on studies of development and regeneration of the central nervous system. This group is identifying the molecular and cellular mechanisms underlying normal brain development and applications in developing novel therapeutic strategies for neurologic disorders including cancer.
The Cincinnati Children's Developmental Neurobiology Research faculty lead discovery programs focused on studies of the neural tumor stem cell and signaling, and include:
- Nadean Brown, PhD: Transcription factors required for specification of retinal progenitor cells into neurons
- Kenneth Campbell, PhD: Cellular and molecular control of vertebrate forebrain development
- Chiou-Fen Chuang, PhD: Molecular mechanisms of neural circuit development by gap junction networks
- Tiffany Cook, PhD: Molecular basis of eye development
- Brian Gebelein, PhD: Patterning of the nervous and digestive systems during development
- Chia-Yi Kuan, MD, PhD: Regulation of neuronal cell death and neurogenesis during development and in ischemic brain injury
- David Repaske, MD, PhD: Molecular basis of endocrine disorders; control of signal transduction
- Randy Sallee, MD, PhD: Molecular pharmacology and pharmacogenetics in neuropsychiatric disorders
- Charles Vorhees, PhD: Mechanism of drug addition and learning and memory
- Yutaka Yoshida, PhD: Molecular basis of sensory-motor connectivity
These associations between the laboratory and academic clinical investigators have provided a platform for rapid translation of new advances in cancer biology and cancer targeting into treatments for patients.
In related translational work published in the January 2008 New England Journal of Medicine, Cincinnati Children's investigators John Bissler and David Franz observed that pharmacologic inhibition of pathologically activated mTOR with rapamycin in tuberous sclerosis complex or sporadic lymphangioleiomyomatosis was associated with regression of angiomyolipomas and improvements in pulmonary function.
